Overview

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor

Status:
Completed
Trial end date:
2019-05-03
Target enrollment:
0
Participant gender:
All
Summary
The purpose of this study is to assess the feasibility of Plerixafor used in combination with G-CSF (Granulocyte Colony Stimulating Factor) in 5 Fanconi anemia patients to mobilize and collect a sufficient number of peripheral blood CD34+ cells for peripheral blood apheresis, for further gene therapy study.
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborator:
EuroFancolen
Treatments:
JM 3100
Lenograstim
Plerixafor
Criteria
Inclusion Criteria:

- Patient with Fanconi anemia

- Patient from 2 to 17 years old

- Potential indication for allogenic bone arrow graft without HLA-identical brotherhood
available

- Patient's weight >10kg

- Treated and followed for at least the previous two years in a specialized center where
they got a full assessment of their disease

- For women of childbearing age, not pregnant and use of an effective contraception
during the entire participation in the research.

- Affiliated or beneficiary of an health insurance regimen

- Informed and signed consent

Exclusion Criteria:

- Patient unable to follow the visits required by the protocol

- Positive serology for HIV-1/2, HTLV-1/2, HCV and HbS

- Bacterial, viral, fungal or parasitic active infection with clinical signs

- Personal history of cancer, myeloproliferative hematopathy or immune deficiency

- Heart failure and / or heart rhythm disorder

- History of allogeneic graft of hematopoietic stem cells

- Patient with an HLA-identical brotherhood donor available

- Myelodysplasia diagnose on myelogram

- Cytogenetic abnormality on karyotype

- Malignant solid tumor

- Documented spontaneous genetic reversion of medullary process

- Diagnosis of a psychiatric disorder that could compromise his/her ability to
participate in the study

- Any disorder according to the investigator, that could compromise the ability of
patient to give his writing consent and/or to comply with requiring study's procedures

- Current Pregnancy

- Heart, kidney or liver failure

- Current participation in another interventional clinical trial

- Patient under Medical Assistance State

- Hypersensitivity to plerixafor or any excipient contained in MOZOBIL®

- Hypersensitivity to filgrastim or any of its' excipient